Thursday, November 5, 2020
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Gene therapy treats "bubble babies"

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There is a new hope for the so-called "bubble children", those children with ADA-SCID, a very serious and rare form of hereditary immunodeficiency which makes it easy prey for viruses and bacteria.

There is a new method of treatment, obtained by modifying patient's stem cells by inserting the gene responsible for the disease, revised and corrected. A milestone, called gene therapy, that will allow these children to live in the real world, outside the bubble that protects them day and night.

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Luigi Naldini, director of the San Raffaele Telethon institute for gene therapy, said: “It's like living in a fairy tale. Children who previously died today are saved by returning to a normal life ».

THEADA-SCID it is a very rare disease, caused by the presence of an altered gene inherited from both parents capable of blocking the production of an essential protein for lymphocytes, adenosine deaminase (Ada). Without lymphocytes, children are virtually deprived of the immune system. This new therapy can now give new hope to many families.

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